Biotech Values

[ghmm]

SRPT:

Sorry I am still not following where the harm is coming from approving E. Is it that patients will never take a potentially better drug, D, because of AE profiles? If so how is that different than other areas where 2 (or more) drugs in the same class are approved? Many claim Lipitor was not the best statin but won out because of marketing. In the E vs D debate if one drug ends up showing meaningful benefit I would imagine switching would occur in numbers porportion to magnitude of perceived efficacy improvement. I still believe both drugs should be approved and both likely offer some modest improvement with neither being substantial. I do agree they have different profiles and could see some doing a little better with one over the other but it remains to be seen if that is an easily identifiable population. Still I don't see the harm in approving eteplirsen (w/wo drisapersen being approved).

My point on starting early is the modest benefit that some (I) believe eteplirsen has may become more meaningful with (much) longer and earlier use. So the argument against it not being worthy of approval because it does almost nothing could be proven inaccurate in real world application (thats not practical in clinical trials) with patients taking the drug for life from a young age. The distrophin data while confrontational is the best supportive argument that I know of (at least for Eteplirsen). I imagine its something the agency will consider and likely both companies will present some data to support the argument of long term/early use improving outcomes.